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FDA授予百特pacritinib快车道地位

来源:生物谷 2014-08-08 14:38

2014年8月8日讯 /生物谷BIOON/ --百特国际(Baxter International)和合作伙伴CTI生物制药公司8月7日联合宣布,FDA已授予pacritinib快车道地位,用于治疗中危及高危骨髓纤维化(myelofibrosis,MF),包括但不限于伴有血小板减少症的患者、正经历其他JAK2疗法出现血小板减少症的患者、以及对其他JAK2疗法不耐受或其症状经其他JAK2疗法管理不佳的患者。目前,pacritinib正处于2个III期临床(即PERSIST项目),评估用于骨髓纤维化患者的治疗。

Pacritinib是一种口服酪氨酸激酶抑制剂,具有针对JAK2和FLT3的双重活性。JAK激酶家族是信号传导通路的重要组成部分,对于正常血细胞的生长和发育、炎性细胞因子的表达、免疫反应至关重要。已有研究表明,这些激酶的突变与各类血液相关癌症的形成直接相关,包括骨髓增生性肿瘤、白血病和淋巴瘤。Pacritinib能够有效治疗疾病症状,同时具有更少的药物出现的血小板减少症及贫血,这些副作用常见于目前已获批及在研的JAK抑制剂;因此,与其他JAK抑制剂相比,pacritinib具有很大的优势。

骨髓纤维化(myelofibrosis,MF)简称髓纤,是一种由于骨髓造血组织中胶原增生,其纤维组织严重地影响造血功能所引起的一种骨髓增生性疾病。骨髓纤维化是由恶性骨髓细胞的积累所致,触发炎症反应,使骨髓形成瘢痕,限制骨髓产生红细胞的能力,并促使脾脏和肝脏接管造血功能。该病具有不同程度的骨髓纤维组织增生,以及主要发生在脾、其次在肝和淋巴结内的髓外造血,典型的临床表现为幼红细胞及幼粒细胞性贫血,并有较多的泪滴状红细胞,骨髓穿刺常出现干抽,脾常明显肿大,并具有不同程度的骨质硬化。

CTI与百特国际(Baxter International)于2013年11月达成全球授权,合作开发和商业化pacritinib,CTI和百特将在美国联合商业化pacritinib,而百特拥有该药在美国以外地区所有适应症的独家商业化权利。(生物谷Bioon.com)

英文原文:FDA Grants Fast Track Designation to CTI BioPharma's Pacritinib, a Novel JAK2 Inhibitor for the Treatment of Myelofibrosis

SEATTLE, Aug. 7, 2014 --CTI BioPharma Corp. (CTI or the Company) (NASDAQ and MTA: CTIC) announced today that pacritinib has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of intermediate and high risk myelofibrosis, including but not limited to patients with disease related thrombocytopenia, patients experiencing treatment emergent thrombocytopenia on other JAK2 therapy or patients who are intolerant to or whose symptoms are sub-optimally managed on other JAK2 therapy. Pacritinib is an oral tyrosine kinase inhibitor with dual activity against JAK2 and FLT3. The drug candidate is currently being evaluated in two Phase 3 clinical trials, known as the PERSIST program, for patients with myelofibrosis.

"We are very pleased that the pacritinib development program in myelofibrosis has been granted Fast Track designation, and we look forward to continuing to work closely with the FDA on this important drug candidate," stated James A. Bianco, M.D., President and CEO. "We believe that pacritinib's unique profile has the potential to serve an unmet medical need that currently exists in this patient population, particularly for those patients with disease or therapy-related low platelet counts."

The Fast Track process is designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need. An unmet need is a condition whose treatment or diagnosis is not addressed adequately by available therapy. The purpose of the Fast Track designation is to make important new drugs available to the patient earlier. The Fast Track program also enables a company to submit sections of the NDA on a rolling basis as data becomes available. This enables the FDA to review sections of the NDA as they are received, rather than waiting until every section of the application is completed before the entire application can be reviewed. NDA review usually does not begin until the company has submitted the entire application to the FDA. A drug program with Fast Track designation enables the company to have early and frequent communication with the FDA in the development and review of the product candidate, often leading to faster drug approval and access by patients.

About the PERSIST Phase 3 Development Program of Pacritinib

Based on pacritinib's efficacy and tolerability profile demonstrated to date, CTI is pursuing a broad approach to advancing this therapy for patients with myelofibrosis by conducting two Phase 3 clinical trials: one in a broad set of patients without limitations on blood platelet counts, the PERSIST-1 trial, and the other in patients with low platelet counts, the PERSIST-2 trial.

PERSIST-1:
In July 2014, CTI completed enrollment in the PERSIST-1 trial that was designed to enroll approximately 320 patients and is a randomized, open-label, multicenter trial comparing the efficacy and safety of pacritinib with that of best available therapy, other than JAK inhibitors, in patients with primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis, without exclusion for low platelet counts. The primary endpoint is the percentage of patients achieving a greater than or equal to 35 percent reduction in spleen volume measured by MRI or CT from baseline to 24 weeks of treatment.

PERSIST-2:
In March 2014, CTI announced the initiation of the PERSIST-2 trial, which will evaluate pacritinib compared to best available therapy, including approved JAK2 inhibitors that are dosed according to product label, in patients with myelofibrosis whose platelet counts are less than or equal to 100,000/uL. The trial is designed to enroll up to 300 patients in North America, Europe, Australia and New Zealand. In October 2013, CTI reached agreement with the FDA on a Special Protocol Assessment (SPA) for the PERSIST-2 trial, which is a written agreement between CTI and the FDA regarding the planned design, endpoints and statistical analysis approach of the trial to be used in support of a potential NDA submission. Under the SPA, the agreed upon co-primary endpoints are the percentage of patients achieving a 35 percent or greater reduction in spleen volume measured by MRI or CT scan from baseline to 24 weeks of treatment and the percentage of patients achieving a Total Symptom Score (TSS) reduction of 50 percent or greater using six key symptoms as measured by the modified Myeloproliferative Neoplasm Symptom Assessment (MPN-SAF TSS 2.0) diary from baseline to 24 weeks.

More details on the PERSIST-1 and PERSIST-2 trials can be found at www.clinicaltrials.gov.

About Pacritinib

Pacritinib is an oral tyrosine kinase inhibitor with dual activity against JAK2 and FLT3. The JAK family of enzymes is a central component in signal transduction pathways, which are critical to normal blood cell growth and development, as well as inflammatory cytokine expression and immune responses. Mutations in these kinases have been shown to be directly related to the development of a variety of blood-related cancers, including myeloproliferative neoplasms, leukemia and lymphoma. Pacritinib may offer an advantage over other JAK inhibitors through effective treatment of symptoms while having less treatment-emergent thrombocytopenia and anemia than has been seen in currently approved and in-development JAK inhibitors.

In November 2013, CTI and Baxter International (Baxter) entered into a worldwide license agreement to develop and commercialize pacritinib in which CTI and Baxter will jointly commercialize pacritinib in the U.S. and Baxter has exclusive commercialization rights for all indications outside the U.S.

About Myelofibrosis

Myelofibrosis is classified as a myeloproliferative neoplasm and is a chronic bone marrow disorder. Myelofibrosis is caused by the accumulation of malignant bone marrow cells that triggers an inflammatory response, scarring the bone marrow and limiting its ability to produce red blood cells, prompting the spleen and liver to take over this function. Symptoms that arise from this disease include enlargement of the spleen, anemia, extreme fatigue and pain. 

About CTI BioPharma

CTI BioPharma Corp. (NASDAQ and MTA: CTIC) is a biopharmaceutical company focused on the acquisition, development and commercialization of novel targeted therapies covering a spectrum of blood-related cancers that offer a unique benefit to patients and healthcare providers. CTI has a commercial presence in Europe and a late-stage development pipeline, including pacritinib, CTI's lead product candidate that is currently being studied in a Phase 3 program for the treatment of patients with myelofibrosis. CTI is headquartered in Seattle, Washington, with offices in London and Milan under the name CTI Life Sciences Limited.

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